Curing Genetic Disease before Birth via Genome Editing

Scientists reported on Monday that they used the genome editing technique CRISPR to alter the DNA of laboratory mice in the womb, eliminating an often-fatal liver disease before the animals had even been born.

For their study, published in Nature Medicine, Musunuru and his colleagues gently opened the uterus of a pregnant mouse, removed the fetus from the amniotic sac, and injected CRISPR into the vitelline vein, which is near the surface of the sac and connects to the liver. “We wanted to make sure we got the genome editor into the liver rather than everywhere else,” Musunuru said. The fetus was then replaced in the uterus and was born normally.

The scientists hope to study fetal base editing for other severe congenital diseases. It remains to be seen whether this technique or conventional gene therapy, which provides an entire replacement gene, will work better. “I’d consider that CRISPR isn’t a replacement” for the latter, Waddington said, “but will be an additional tool” for curing genetic diseases in the womb.

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